“Children and adults with late onset LAL Deficiency can
suffer from serious liver complications and accelerated
atherosclerosis,” said Manisha Balwani, MD, MS, Assistant
Professor of Genetics and Genomic Sciences and Assistant Professor
of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY,
and principal investigator in the study. “Based on its
mechanism of action, sebelipase alfa addresses the root cause of
LAL Deficiency, and the Phase I/II extension study in adults with
LAL Deficiency illustrates that sebelipase alfa improves patients'
abnormalities associated with the disease.”

About the Phase I/II extension study of sebelipase alfa in
adults with late onset LAL Deficiency

Nine adults with LAL Deficiency were enrolled in the Phase I/II
trial. After completing the initial portion of the Phase I/II
trial, patients were allowed to continue treatment with sebelipase
alfa as part of a long-term, open-label extension study.

In the extension study, patients received four once-weekly
infusions of sebelipase alfa (0.35 mg/kg, 1.0 mg/kg, or 3.0 mg/kg)
and then transitioned to every other week infusions of sebelipase
alfa (1.0 mg/kg or 3.0 mg/kg). Eight of nine patients enrolled in
the extension study. Data published in Hepatology were
derived from the seven patients who completed the first 12 weeks of
dosing in the extension study.

For these seven patients, sebelipase alfa produced mean percent
decreases for ALT and AST from the initial baseline to week 12 of
the extension study of 52% and 36%, respectively (p<0.05 for
both). In addition, sebelipase alfa resulted in mean percent
decreases from the initial baseline to week 12 of the extension
study for LDL-C of 27% (p=0.078), total cholesterol of 22%
(p=0.047), triglycerides of 28% (p=0.016), as well as mean
increases in HDL of 15% (p=0.016).

Patients in the Phase I/II trial had a mean age of 32 years
(range 19-45); two-thirds were male. Eight of the nine patients had
evidence of hepatomegaly on clinical exam, and two of the nine
patients had evidence of more advanced liver disease, including
cirrhosis and portal hypertension. Seven patients had a history of
other cardiovascular conditions. Seven of the nine patients were
receiving lipid modifying therapies including ezetimibe, statins,
and other medications.

Sebelipase alfa was well tolerated throughout the initial 12
weeks of the extension study. The majority of adverse events were
mild and unrelated to sebelipase alfa. Infusion-related reactions
were uncommon and the majority were gastrointestinal (diarrhea,
abdominal cramping) events of mild severity. No anti-drug
antibodies were detected in any subjects in either the initial
portion or the 12-week extension portion of the Phase I/II study. A
single patient during the extension study developed acute
cholecystitis and cholelithiasis (two serious adverse events) which
were later treated with elective cholecystectomy. These two serious
adverse events were considered unlikely related to sebelipase alfa.
This patient has continued treatment with sebelipase alfa without
changes in dosing and administration.

“The publication of these data in Hepatology
underscores the ability of sebelipase alfa to reduce markers of
liver damage and improve dyslipidemia in these adults with LAL
Deficiency,” said Anthony Quinn, MBChB, PhD, FRCP, Senior
Vice President and Chief Medical Officer of Synageva BioPharma.
“Along with the Phase III trial in children and adults with
late onset LAL Deficiency, this publication will also help support
efforts to raise awareness for a disease that remains an
underappreciated cause of cirrhosis in children and
adults.”

About Synageva's Lead Program

Sebelipase alfa (formerly referred to as SBC-102) is a
recombinant form of the human LAL enzyme under development by
Synageva as an enzyme replacement therapy for LAL Deficiency, a
lysosomal storage disorder (LSD). Synageva is currently evaluating
sebelipase alfa in global clinical trials for both early and late
onset LAL Deficiency. Sebelipase alfa has been granted orphan
designations by the U.S. Food and Drug Administration (FDA), the
European Medicines Agency, and the Japanese Ministry of Health,
Labour and Welfare. Additionally, sebelipase alfa received
“fast track” designation by the FDA.

About LAL Deficiency

LAL Deficiency is a rare autosomal recessive LSD caused by a
marked decrease in LAL enzyme activity. Late onset LAL Deficiency,
sometimes called Cholesteryl Ester Storage Disease (CESD), affects
both children and adults. In these patients, the buildup of fatty
material in the liver and blood vessel walls may lead to liver
cirrhosis, liver failure and accelerated atherosclerotic events.
Early onset LAL Deficiency, sometimes called Wolman disease,
affects infants and is characterized by severe malabsorption,
growth failure and liver failure, and is usually fatal within the
first six months of life. There are no approved pharmacological
therapies for LAL Deficiency. Success with stem cell and liver
transplant appears to be limited by procedure-related morbidity and
mortality.

About Synageva BioPharma Corp.

Synageva is a clinical stage biopharmaceutical company focused
on the discovery, development, and commercialization of therapeutic
products for patients with life-threatening rare diseases and unmet
medical need. Synageva has several protein therapeutics in its drug
development pipeline. The company has assembled a team with a
proven record of bringing therapies to patients with rare
diseases.

Further information regarding Synageva BioPharma Corp. is
available at
www.synageva.com.

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